Am J Respir Crit Care Med. 2021 Oct 15;204(8):891-901. doi: 10.1164/rccm.202108-1908ST.
Precision medicine focuses on the identification of therapeutic strategies that are effective for a group of patients based on similar unifying characteristics. The recent success of precision medicine in non-critical care settings has resulted from the confluence of large clinical and biospecimen repositories, innovative bioinformatics, and novel trial designs. Similar advances for precision medicine in sepsis and in the acute respiratory distress syndrome (ARDS) are possible but will require further investigation and significant investment in infrastructure. This project was funded by the American Thoracic Society Board of Directors. A multidisciplinary and diverse working group reviewed the available literature, established a conceptual framework, and iteratively developed recommendations for the Precision Medicine Research Agenda for Sepsis and ARDS. The following six priority recommendations were developed by the working group: ) the creation of large richly phenotyped and harmonized knowledge networks of clinical, imaging, and multianalyte molecular data for sepsis and ARDS; ) the implementation of novel trial designs, including adaptive designs, and embedding trial procedures in the electronic health record; ) continued innovation in the data science and engineering methods required to identify heterogeneity of treatment effect; ) further development of the tools necessary for the real-time application of precision medicine approaches; ) work to ensure that precision medicine strategies are applicable and available to a broad range of patients varying across differing racial, ethnic, socioeconomic, and demographic groups; and ) the securement and maintenance of adequate and sustainable funding for precision medicine efforts. Precision medicine approaches that incorporate variability in genomic, biologic, and environmental factors may provide a path forward for better individualizing the delivery of therapies and improving care for patients with sepsis and ARDS.
精准医学侧重于根据相似的统一特征,确定对一组患者有效的治疗策略。最近,在非重症监护环境中,精准医学的成功源于大型临床和生物样本库、创新的生物信息学和新型试验设计的融合。在脓毒症和急性呼吸窘迫综合征(ARDS)中,类似的精准医学进展是可能的,但需要进一步的研究和对基础设施的大量投资。 本项目由美国胸科学会董事会资助。一个多学科和多元化的工作组审查了现有文献,建立了一个概念框架,并为脓毒症和 ARDS 的精准医学研究议程迭代开发了建议。工作组制定了以下六项优先建议: )创建大型、丰富表型和协调的临床、影像和多分析物分子数据的知识网络,用于脓毒症和 ARDS; )实施新型试验设计,包括适应性设计,并将试验程序嵌入电子健康记录中; )继续创新识别治疗效果异质性所需的数据科学和工程方法; )进一步开发实时应用精准医学方法所需的工具; )努力确保精准医学策略适用于并可用于广泛的患者,这些患者在不同的种族、民族、社会经济和人口统计学群体中有所不同;以及 )确保精准医学工作的充足和可持续资金。 纳入基因组、生物学和环境因素变异性的精准医学方法可能为更好地个体化治疗提供途径,并改善脓毒症和 ARDS 患者的护理。